Publications

Publications and Results

Hautbergue, G.M., Castelli, L.M, & Ferraiuolo, L. Et al. (2017) SRSF1-dependent nuclear export inhibition of C9ORF72 repeat transcripts prevents neurodegeneration and associated motor deficits. Nature Communication. 5:8:16063.doi: 10.1038

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Castelli, L.M, Cutillo, L., Dos Santos Souza, C., et al SRSF1-dependent inhibition of C9ORF72-repeat RNA nuclear export: genome-wide mechanisms for neuroprotection in amyotrophic lateral sclerosis. Castelli et al. Molecular Neurodegeneration 2021;16(1): 53

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Castelli, L.M, Lin, Y-H., Sachez-Martinez, A., et al A cell-penetrant peptide blocking C9ORF72-repeat RNA nuclear export reduces the neurotoxic effects of dipeptide repeat proteins. Science Translational Medicine 2023; 15, eabo3823

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Mizielinska S, Hautbergue GM, et al. Amyotrophic lateral sclerosis caused by hexanucleotide repeat expansions in C9orf72: from genetics to therapeutics. Invited review. Lancet Neurology 2025; 24(3): 261-274

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